EP2609198B8 - SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER - Google Patents

SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER Download PDF

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Publication number
EP2609198B8
EP2609198B8 EP11820420.5A EP11820420A EP2609198B8 EP 2609198 B8 EP2609198 B8 EP 2609198B8 EP 11820420 A EP11820420 A EP 11820420A EP 2609198 B8 EP2609198 B8 EP 2609198B8
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EP
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Prior art keywords
internal
nucleic acid
agents containing
rnai agents
stranded rnai
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Application number
EP11820420.5A
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German (de)
English (en)
French (fr)
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EP2609198A4 (en
EP2609198A1 (en
EP2609198B1 (en
Inventor
Lee Lim
Aarron Willingham
Lihong Zhao
Guillaume Chorn
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Sirna Therapeutics Inc
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Sirna Therapeutics Inc
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Priority to EP18000072.1A priority Critical patent/EP3372684B1/en
Publication of EP2609198A1 publication Critical patent/EP2609198A1/en
Publication of EP2609198A4 publication Critical patent/EP2609198A4/en
Publication of EP2609198B1 publication Critical patent/EP2609198B1/en
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • C12N2310/141MicroRNAs, miRNAs
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183Diol linkers, e.g. glycols or propanediols
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
EP11820420.5A 2010-08-24 2011-08-19 SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER Active EP2609198B8 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP18000072.1A EP3372684B1 (en) 2010-08-24 2011-08-19 Single-stranded rnai agents containing an internal, non-nucleic acid spacer

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US37647110P 2010-08-24 2010-08-24
PCT/US2011/048338 WO2012027206A1 (en) 2010-08-24 2011-08-19 SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER

Related Child Applications (2)

Application Number Title Priority Date Filing Date
EP18000072.1A Division EP3372684B1 (en) 2010-08-24 2011-08-19 Single-stranded rnai agents containing an internal, non-nucleic acid spacer
EP18000072.1A Division-Into EP3372684B1 (en) 2010-08-24 2011-08-19 Single-stranded rnai agents containing an internal, non-nucleic acid spacer

Publications (4)

Publication Number Publication Date
EP2609198A1 EP2609198A1 (en) 2013-07-03
EP2609198A4 EP2609198A4 (en) 2014-11-19
EP2609198B1 EP2609198B1 (en) 2018-02-21
EP2609198B8 true EP2609198B8 (en) 2018-03-28

Family

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Family Applications (2)

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EP18000072.1A Active EP3372684B1 (en) 2010-08-24 2011-08-19 Single-stranded rnai agents containing an internal, non-nucleic acid spacer
EP11820420.5A Active EP2609198B8 (en) 2010-08-24 2011-08-19 SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER

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EP18000072.1A Active EP3372684B1 (en) 2010-08-24 2011-08-19 Single-stranded rnai agents containing an internal, non-nucleic acid spacer

Country Status (6)

Country Link
US (3) US9243246B2 (ko)
EP (2) EP3372684B1 (ko)
JP (5) JP6106085B2 (ko)
KR (1) KR20130137160A (ko)
CN (1) CN103140582A (ko)
WO (1) WO2012027206A1 (ko)

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US9243246B2 (en) * 2010-08-24 2016-01-26 Sirna Therapeutics, Inc. Single-stranded RNAi agents containing an internal, non-nucleic acid spacer
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